CA2991109A1 - Compositions et procedes d'administration d'outils d'edition de gene au moyen de vesicules de polymere - Google Patents
Compositions et procedes d'administration d'outils d'edition de gene au moyen de vesicules de polymere Download PDFInfo
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- CA2991109A1 CA2991109A1 CA2991109A CA2991109A CA2991109A1 CA 2991109 A1 CA2991109 A1 CA 2991109A1 CA 2991109 A CA2991109 A CA 2991109A CA 2991109 A CA2991109 A CA 2991109A CA 2991109 A1 CA2991109 A1 CA 2991109A1
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- gene editing
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- HDZZVAMISRMYHH-KCGFPETGSA-N tubercidin Chemical compound C1=CC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O HDZZVAMISRMYHH-KCGFPETGSA-N 0.000 description 1
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- DRTQHJPVMGBUCF-UHFFFAOYSA-N uracil arabinoside Natural products OC1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-UHFFFAOYSA-N 0.000 description 1
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Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/5005—Wall or coating material
- A61K9/5021—Organic macromolecular compounds
- A61K9/5031—Organic macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, poly(lactide-co-glycolide)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Liposomes
- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
- A61K9/1273—Polymersomes; Liposomes with polymerisable or polymerised bilayer-forming substances
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases RNAses, DNAses
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Dispersion Chemistry (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Enzymes And Modification Thereof (AREA)
- Polyesters Or Polycarbonates (AREA)
Abstract
La présente invention concerne une composition pour modification génétique et un procédé de formation de la composition, la composition pouvant comprendre une vésicule de polymère synthétique, et un système d'édition de gène encapsulé dans la vésicule de polymère synthétique. Le système d'édition de gène peut comprendre un composant protéique et un composant d'acide nucléique configuré pour interagir avec une séquence cible dans un génome de cellule hôte.
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562187942P | 2015-07-02 | 2015-07-02 | |
| US62/187,942 | 2015-07-02 | ||
| US201662322346P | 2016-04-14 | 2016-04-14 | |
| US62/322,346 | 2016-04-14 | ||
| US15/199,021 US20170000743A1 (en) | 2015-07-02 | 2016-06-30 | Compositions and Methods for Delivery of Gene Editing Tools Using Polymeric Vesicles |
| US15/199,021 | 2016-06-30 | ||
| PCT/US2016/040673 WO2017004509A1 (fr) | 2015-07-02 | 2016-07-01 | Compositions et procédés d'administration d'outils d'édition de gène au moyen de vésicules de polymère |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA2991109A1 true CA2991109A1 (fr) | 2017-01-05 |
Family
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| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2991109A Pending CA2991109A1 (fr) | 2015-07-02 | 2016-07-01 | Compositions et procedes d'administration d'outils d'edition de gene au moyen de vesicules de polymere |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20170000743A1 (fr) |
| EP (1) | EP3317414A4 (fr) |
| JP (2) | JP6993966B2 (fr) |
| CN (1) | CN108699563A (fr) |
| AU (1) | AU2016288237B2 (fr) |
| CA (1) | CA2991109A1 (fr) |
| HK (1) | HK1255209A1 (fr) |
| WO (1) | WO2017004509A1 (fr) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10456452B2 (en) | 2015-07-02 | 2019-10-29 | Poseida Therapeutics, Inc. | Compositions and methods for improved encapsulation of functional proteins in polymeric vesicles |
| ES2865481T3 (es) * | 2016-04-29 | 2021-10-15 | Poseida Therapeutics Inc | Micelas basadas en poli(histidina) para la complejación y el aporte de proteínas y ácidos nucleicos |
| EP3519561A1 (fr) | 2016-09-30 | 2019-08-07 | Poseida Therapeutics, Inc. | Cellules t de mémoire de cellules souches modifiées, procédés de fabrication et procédés d'utilisation correspondants |
| US20190119636A1 (en) | 2017-10-23 | 2019-04-25 | Poseida Therapeutics, Inc. | Modified stem cell memory t cells, methods of making and methods of using same |
| WO2018068022A1 (fr) | 2016-10-06 | 2018-04-12 | Poseida Therapeutics, Inc. | Caspases inductibles et procédés d'utilisation |
| US20200384130A1 (en) | 2017-06-14 | 2020-12-10 | Dana-Farber Cancer Institute, Inc. | B-cell maturation antigen (bcma)-directed nanoparticles |
| CA3067382A1 (fr) | 2017-06-15 | 2018-12-20 | The Regents Of The University Of California | Insertions ciblees d'adn non viral |
| US10329543B2 (en) | 2017-10-23 | 2019-06-25 | Poseida Therapeutics, Inc. | Modified stem cell memory T cells, methods of making and methods of using same |
| BR112020008201A2 (pt) | 2017-10-27 | 2020-10-06 | The Regents Of The University Of California | substituição-alvo de receptores de células t endógenos |
| EP3898993A1 (fr) | 2018-12-20 | 2021-10-27 | Poseida Therapeutics, Inc. | Compositions de nanotransposons et procédés d'utilisation |
| EP4025593A1 (fr) | 2019-09-05 | 2022-07-13 | Poseida Therapeutics, Inc. | Compositions de cellules allogéniques et procédés d'utilisation |
| WO2021127505A1 (fr) | 2019-12-20 | 2021-06-24 | Poseida Therapeutics, Inc. | Compositions anti-muc1 et méthodes d'utilisation |
| US20230121433A1 (en) | 2020-03-11 | 2023-04-20 | Poseida Therapeutics, Inc. | Chimeric stimulatory receptors and methods of use in t cell activation and differentiation |
| GB202004254D0 (en) | 2020-03-24 | 2020-05-06 | Puridify Ltd | Characterization of gene therapy vectors |
| US20240060090A1 (en) | 2021-02-23 | 2024-02-22 | Poseida Therapeutics, Inc. | Genetically modified induced pluripotent stem cells and methods of use thereof |
| AU2022257004A1 (en) * | 2021-04-13 | 2023-11-23 | Arsenal Biosciences, Inc. | Non-viral homology mediated end joining |
| WO2023060088A1 (fr) | 2021-10-04 | 2023-04-13 | Poseida Therapeutics, Inc. | Compositions de transposons et leurs procédés d'utilisation |
| WO2023164573A1 (fr) | 2022-02-23 | 2023-08-31 | Poseida Therapeutics, Inc. | Cellules modifiées et leurs procédés d'utilisation |
| WO2023193665A1 (fr) * | 2022-04-07 | 2023-10-12 | 威海纽兰生物科技有限公司 | Vecteur pour l'administration de médicament sensible à l'estérase à base de vésicule extracellulaire, son procédé de préparation et son utilisation |
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| AU2001261732A1 (en) | 2000-05-19 | 2001-12-03 | Betsy T. Kren | Composition for delivery of compounds to cells |
| US20040265835A1 (en) * | 2003-06-30 | 2004-12-30 | Lemaster David M. | Method of sorting vesicle-entrapped, coupled nucleic acid-protein displays |
| CA2756670A1 (fr) * | 2009-03-26 | 2010-09-30 | The Regents Of The University Of California | Cellules souches mesenchymateuses produisant de l'arn inhibiteur pouvant etre utilisees pour agir sur le cours d'une maladie |
| KR101701534B1 (ko) * | 2009-04-07 | 2017-02-01 | 다우 아그로사이언시즈 엘엘씨 | 서열 특이적 뉴클레아제의 나노입자 매개 전달 |
| WO2011106376A2 (fr) * | 2010-02-23 | 2011-09-01 | The General Hospital Corporation | Utilisation de microvésicules dans le traitement d'affections médicales |
| WO2011109512A1 (fr) * | 2010-03-02 | 2011-09-09 | Vindico Nanobio Technology, Inc. | Compositions et méthodes de traitement prophylactique ou thérapeutique d'une pathologie immuno-inflammatoire |
| US20140363496A1 (en) * | 2011-01-07 | 2014-12-11 | Vindico NanoBio Technology Inc. | Compositions and Methods for Inducing Nanoparticle-mediated Microvascular Embolization of Tumors |
| BR112013025567B1 (pt) * | 2011-04-27 | 2021-09-21 | Amyris, Inc | Métodos para modificação genômica |
| SG10201707569YA (en) | 2012-12-12 | 2017-10-30 | Broad Inst Inc | Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications |
| JP6552965B2 (ja) | 2012-12-12 | 2019-07-31 | ザ・ブロード・インスティテュート・インコーポレイテッド | 配列操作のための改善された系、方法および酵素組成物のエンジニアリングおよび最適化 |
| WO2014150624A1 (fr) * | 2013-03-14 | 2014-09-25 | Caribou Biosciences, Inc. | Compositions et procédés pour des acides nucléiques à ciblage d'acide nucléique |
| US20150067922A1 (en) * | 2013-05-30 | 2015-03-05 | The Penn State Research Foundation | Gene targeting and genetic modification of plants via rna-guided genome editing |
| CA2951707A1 (fr) * | 2014-06-10 | 2015-12-17 | Massachusetts Institute Of Technology | Procede d'edition genique |
-
2016
- 2016-06-30 US US15/199,021 patent/US20170000743A1/en not_active Abandoned
- 2016-07-01 WO PCT/US2016/040673 patent/WO2017004509A1/fr active Application Filing
- 2016-07-01 EP EP16818879.5A patent/EP3317414A4/fr active Pending
- 2016-07-01 CA CA2991109A patent/CA2991109A1/fr active Pending
- 2016-07-01 AU AU2016288237A patent/AU2016288237B2/en active Active
- 2016-07-01 JP JP2018520385A patent/JP6993966B2/ja active Active
- 2016-07-01 CN CN201680050998.3A patent/CN108699563A/zh active Pending
-
2018
- 2018-11-09 HK HK18114337.3A patent/HK1255209A1/zh unknown
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2019
- 2019-06-05 JP JP2019105213A patent/JP2019165745A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP6993966B2 (ja) | 2022-02-04 |
| HK1255209A1 (zh) | 2019-08-09 |
| AU2016288237B2 (en) | 2022-04-14 |
| CN108699563A (zh) | 2018-10-23 |
| EP3317414A1 (fr) | 2018-05-09 |
| JP2019165745A (ja) | 2019-10-03 |
| AU2016288237A1 (en) | 2018-01-25 |
| EP3317414A4 (fr) | 2019-03-13 |
| US20170000743A1 (en) | 2017-01-05 |
| WO2017004509A1 (fr) | 2017-01-05 |
| JP2018532408A (ja) | 2018-11-08 |
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